20 assets in Spinal Muscular Atrophy

Therapeutic assets addressing Spinal Muscular Atrophy, rated under Floatz methodology v0.2 and sorted by current rating.

Asset	Sponsor	Phase	Rating
Nusinersen Spinal Muscular Atrophy	Scholar Rock, Inc.	P4	A (82)
Risdiplam Spinal Muscular Atrophy	Scholar Rock, Inc.	P4	A (80)
Non-Replicating Recombinant Self-Complementary (Sc) Adeno-Associated Virus Serotype 9 (Aav9) Vector Containing The Cdna Of The Survival Of Motor Neuron 2 (Smn2) Gene Under The Control Of The Hybrid Cytomegalovirus (Cmv) Enhancer/Chicken Beta-Actin Promoter (Cba) Spinal Muscular Atrophy	Novartis Pharmaceuticals	P4	BBB (78)
oav101 Spinal Muscular Atrophy	Novartis Pharmaceuticals	P4	BBB (78)
Apitegromab Spinal Muscular Atrophy	Scholar Rock, Inc.	P3	BB (67)
Vesemnogene Lantuparvovec Spinal Muscular Atrophy	Lantu Biopharma	P3	BB (64)
All-P-Ambo-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thiocytidylyl-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]Adenylyl-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Cytidylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-[(5-Methyl)Uridylyl-)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Cytidylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioadenylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioadenylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioadenylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioguanylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Cytidylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioguanylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioguanylyl)]-(5-Methyl)Cytidine Spinal Muscular Atrophy	Biogen	P3	B (60)
gc101 adeno-associated virus Spinal Muscular Atrophy	GeneCradle Inc	P3	B (60)
Taldefgrobep Alfa Spinal Muscular Atrophy	Biohaven Pharmaceuticals, Inc.	P3	B (60)
Immunoglobulin G1-Kappa, Anti-[Homo Sapiens Mstn (Myostatin, Growth Differentiation Factor 8, Gdf8, Gdf-8) Latent Myostatin Complex], Humanized Monoclonal Antibody; Spinal Muscular Atrophy	Hoffmann-La Roche	P2/3	B (57)
nmd670 Spinal Muscular Atrophy	NMD Pharma A/S	P2	B (57)
Argx 119 Spinal Muscular Atrophy	argenx	P2	CCC (53)
Anb-004 Spinal Muscular Atrophy	Biocad	P1/2	CC (47)
gc101 Spinal Muscular Atrophy	GeneCradle Inc	P1/2	CC (47)
Biib115 Spinal Muscular Atrophy	Biogen	P1	CC (43)
exg001-307 Spinal Muscular Atrophy	Guangzhou Jiayin Biotech Ltd	Early P1	C (39)
Gip Spinal Muscular Atrophy	University of Copenhagen	N/A	D (30)
Glucagon-Like Peptide Ii Spinal Muscular Atrophy	University of Copenhagen	N/A	D (30)
nkg001 Spinal Muscular Atrophy	Nikegen Pharmaceutical (Hangzhou) Company Limited	N/A	D (30)